Targeted exon skipping with AAV-mediated split adenine base editors-Reference-Cited by-同舟云学术

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Targeted exon skipping with AAV-mediated split adenine base editors

Published:2019-08-20 Issue:1 Volume:5 Page:
ISSN:2056-5968
Container-title:Cell Discovery
language:en
Short-container-title:Cell Discov

Author:

Winter Jackson,Luu Alan,Gapinske Michael,Manandhar Sony,Shirguppe Shraddha,Woods Wendy S.,Song Jun S.,Perez-Pinera Pablo

Funder

American Heart Association

U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences

U.S. Department of Health & Human Services | NIH | National Institute of Biomedical Imaging and Bioengineering

National Science Foundation

U.S. Department of Health & Human Services | NIH | National Cancer Institute

Publisher

Springer Science and Business Media LLC

Subject

Cell Biology,Genetics,Molecular Biology,Biochemistry

Link

http://www.nature.com/articles/s41421-019-0109-7.pdf

Reference46 articles.

1. Pan, Q., Shai, O., Lee, L. J., Frey, B. J. & Blencowe, B. J. Deep surveying of alternative splicing complexity in the human transcriptome by high-throughput sequencing. Nat. Genet. 40, 1413–1415 (2008).

2. Kole, R. & Krieg, A. M. Exon skipping therapy for Duchenne muscular dystrophy. Adv. Drug Deliv. Rev. 87, 104–107 (2015).

3. Kowalewski, C. et al. Amelioration of junctional epidermolysis bullosa due to exon skipping. Br. J. Dermatol. 174, 1375–1379 (2016).

4. Burghes, A. H. & McGovern, V. L. Antisense oligonucleotides and spinal muscular atrophy: skipping along. Genes Dev. 24, 1574–1579 (2010).

5. Crooke, S. T. Molecular mechanisms of action of antisense drugs. Biochim. Biophys. Acta 1489, 31–44 (1999).

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